The Developing Regorafenib Eye drops for neovascular Age-related Macular degeneration (DREAM) study: an open-label phase II trial
Joussen AM, Wolf S, Kaiser PK, Boyer D, Schmelter T, Sandbrink R, Zeitz O, Deeg G, Richter A, Zimmermann T, Hoechel J, Buetehorn U, Schmitt W, Stemper B, Boettger MK (2019)
Publication Type: Journal article
Publication year: 2019
Journal
Book Volume: 85
Journal Issue: 2
DOI: 10.1111/bcp.13794
Abstract
Aims This programme investigated topical regorafenib, a multikinase inhibitor, in patients with neovascular age-related macular degeneration (nAMD). Methods Topical regorafenib was investigated in an open-label, phase IIa/b study in which patients with choroidal neovascularization (CNV) secondary to nAMD received regorafenib (25 mu l, 30 mg ml(-1)) three times a day for 12 weeks. The primary endpoint of the phase II/a/b study was mean change in best-corrected visual acuity (BCVA) from baseline to weeks 4 and 12. Results In nAMD patients (N = 51), mean changes in BCVA were +1.2 [90% confidence interval (CI) -0.61, 2.97] and -2.4 (90% CI -4.18, -0.54) letters at weeks 4 and 12, respectively. Ocular treatment-emergent adverse events (TEAEs) (study eye) were reported in 21 patients by week 12. There was one serious ocular TEAE (visual acuity reduced) that was not drug related. Twenty patients required rescue (intravitreal ranibizumab). Conclusions The programme was terminated after phase IIa ended because efficacy was lower than with current nAMD treatments. According to elaborate post hoc analyses, the most likely reason was insufficient exposure in the target compartment (back of the eye).
Additional Organisation(s)
Involved external institutions
Cole Eye Institute
United States (USA) (US)
Bayer AG
Germany (DE)
Charité - Universitätsmedizin Berlin
Germany (DE)
Universität Bern
Switzerland (CH)
Retina-Vitreous Associates Medical Group
United States (USA) (US)
United States (USA) (US)
Bayer AG
Germany (DE)
Charité - Universitätsmedizin Berlin
Germany (DE)
Universität Bern
Switzerland (CH)
Retina-Vitreous Associates Medical Group
United States (USA) (US)
How to cite
APA:
Joussen, A.M., Wolf, S., Kaiser, P.K., Boyer, D., Schmelter, T., Sandbrink, R.,... Boettger, M.K. (2019). The Developing Regorafenib Eye drops for neovascular Age-related Macular degeneration (DREAM) study: an open-label phase II trial. British Journal of Clinical Pharmacology, 85(2). https://dx.doi.org/10.1111/bcp.13794
MLA:
Joussen, Antonia M., et al. "The Developing Regorafenib Eye drops for neovascular Age-related Macular degeneration (DREAM) study: an open-label phase II trial." British Journal of Clinical Pharmacology 85.2 (2019).
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